JP7247227B2 - Ａｐｏｌ１発現のモジュレーター - Google Patents

Ａｐｏｌ１発現のモジュレーター Download PDF

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JP7247227B2
JP7247227B2 JP2020565431A JP2020565431A JP7247227B2 JP 7247227 B2 JP7247227 B2 JP 7247227B2 JP 2020565431 A JP2020565431 A JP 2020565431A JP 2020565431 A JP2020565431 A JP 2020565431A JP 7247227 B2 JP7247227 B2 JP 7247227B2
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certain embodiments
modified
compound
oligonucleotide
apol1
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Japanese (ja)
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JP2021525084A (ja
JPWO2019226611A5 (fr
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スーザン・エム・フレイア
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Priority to JP2023040313A priority Critical patent/JP7645301B2/ja
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Priority to JP2024194079A priority patent/JP2025013513A/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Urology & Nephrology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2020565431A 2018-05-22 2019-05-21 Ａｐｏｌ１発現のモジュレーター Active JP7247227B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023040313A JP7645301B2 (ja) 2018-05-22 2023-03-15 Ａｐｏｌ１発現のモジュレーター
JP2024194079A JP2025013513A (ja) 2018-05-22 2024-11-06 Ａｐｏｌ１発現のモジュレーター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862674865P 2018-05-22 2018-05-22
US62/674,865 2018-05-22
PCT/US2019/033244 WO2019226611A1 (fr) 2018-05-22 2019-05-21 Modulateurs de l'expression d'apol1

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023040313A Division JP7645301B2 (ja) 2018-05-22 2023-03-15 Ａｐｏｌ１発現のモジュレーター

Publications (3)

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JP2021525084A JP2021525084A (ja) 2021-09-24
JPWO2019226611A5 JPWO2019226611A5 (fr) 2022-03-24
JP7247227B2 true JP7247227B2 (ja) 2023-03-28

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JP2020565431A Active JP7247227B2 (ja) 2018-05-22 2019-05-21 Ａｐｏｌ１発現のモジュレーター
JP2023040313A Active JP7645301B2 (ja) 2018-05-22 2023-03-15 Ａｐｏｌ１発現のモジュレーター
JP2024194079A Pending JP2025013513A (ja) 2018-05-22 2024-11-06 Ａｐｏｌ１発現のモジュレーター

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JP2023040313A Active JP7645301B2 (ja) 2018-05-22 2023-03-15 Ａｐｏｌ１発現のモジュレーター
JP2024194079A Pending JP2025013513A (ja) 2018-05-22 2024-11-06 Ａｐｏｌ１発現のモジュレーター

Country Status (27)

Country Link
US (4) US10927377B2 (fr)
EP (1) EP3799570A4 (fr)
JP (3) JP7247227B2 (fr)
KR (2) KR20250153881A (fr)
CN (3) CN112423792B (fr)
AR (1) AR115416A1 (fr)
AU (2) AU2019274461B2 (fr)
BR (1) BR112020023436A2 (fr)
CA (1) CA3099750A1 (fr)
CL (1) CL2020003010A1 (fr)
CO (1) CO2020015377A2 (fr)
CR (1) CR20200631A (fr)
EA (1) EA202092748A1 (fr)
EC (1) ECSP20082339A (fr)
IL (2) IL316397A (fr)
JO (1) JOP20200291A1 (fr)
MA (1) MA53924A (fr)
MX (2) MX2020012497A (fr)
MY (1) MY200352A (fr)
NI (1) NI202000085A (fr)
PE (1) PE20211397A1 (fr)
PH (1) PH12020551995A1 (fr)
SG (1) SG11202011397QA (fr)
TW (2) TW202430638A (fr)
UA (1) UA128819C2 (fr)
WO (1) WO2019226611A1 (fr)
ZA (1) ZA202007537B (fr)

Families Citing this family (9)

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USRE49076E1 (en) 2010-04-18 2022-05-17 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for treating renal disease
IL316397A (en) 2018-05-22 2024-12-01 Ionis Pharmaceuticals Inc Modulators of APOL1 expression
CN113453760B (zh) 2018-12-17 2024-05-24 弗特克斯药品有限公司 Apol1抑制剂及其使用方法
AU2021213758A1 (en) 2020-01-29 2022-09-01 Vertex Pharmaceuticals Incorporated Inhibitors of APOL1 and methods of using same
CA3183591A1 (fr) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Formes solides d'un inhibiteur d'apol1 et utilisation de celles-ci
CN116157393A (zh) 2020-06-12 2023-05-23 弗特克斯药品有限公司 Apol1的抑制剂及其用途
MX2024008868A (es) 2022-01-18 2024-09-23 Maze Therapeutics Inc Inhibidores de la apolipoproteína l1 (apol1) y métodos de uso.
EP4573077A1 (fr) * 2022-08-19 2025-06-25 Maze Therapeutics, Inc. Inhibiteurs d'apol1 et procédés d'utilisation
WO2025242689A1 (fr) 2024-05-21 2025-11-27 Astrazeneca Ab Méthodes de traitement d'une maladie rénale médiée par apol1

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JP2015507924A (ja) 2012-02-08 2015-03-16 アイシス ファーマシューティカルズ， インコーポレーテッド 第ｖｉｉ因子の発現を調節するための方法と組成物
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JP2015507924A (ja) 2012-02-08 2015-03-16 アイシス ファーマシューティカルズ， インコーポレーテッド 第ｖｉｉ因子の発現を調節するための方法と組成物
WO2017053722A1 (fr) 2015-09-24 2017-03-30 Ionis Pharmaceuticals, Inc. Modulateurs de l'expression de kras

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AR115416A1 (es) 2021-01-13
UA128819C2 (uk) 2024-10-30
CN118127020A (zh) 2024-06-04
US10927377B2 (en) 2021-02-23
AU2025259982A1 (en) 2026-01-22
TW202016305A (zh) 2020-05-01
EP3799570A4 (fr) 2022-04-27
KR102874776B1 (ko) 2025-10-24
ECSP20082339A (es) 2021-02-26
MY200352A (en) 2023-12-21
CN112423792A (zh) 2021-02-26
MX2025003294A (es) 2025-05-02
WO2019226611A1 (fr) 2019-11-28
MA53924A (fr) 2021-08-25
US20250066785A1 (en) 2025-02-27
BR112020023436A2 (pt) 2021-02-23
CA3099750A1 (fr) 2019-11-28
CL2020003010A1 (es) 2021-06-11
CR20200631A (es) 2021-02-10
MX2020012497A (es) 2021-02-15
JP2025013513A (ja) 2025-01-24
IL278785A (en) 2021-01-31
JP7645301B2 (ja) 2025-03-13
US11525136B2 (en) 2022-12-13
TW202430638A (zh) 2024-08-01
US20210079395A1 (en) 2021-03-18
CO2020015377A2 (es) 2020-12-21
CN118127019A (zh) 2024-06-04
US20230265428A1 (en) 2023-08-24
AU2019274461B2 (en) 2025-07-31
TWI841564B (zh) 2024-05-11
ZA202007537B (en) 2021-09-29
SG11202011397QA (en) 2020-12-30
US20190359981A1 (en) 2019-11-28
JP2021525084A (ja) 2021-09-24
IL278785B2 (en) 2025-03-01
IL316397A (en) 2024-12-01
JOP20200291A1 (ar) 2020-11-15
JP2023075283A (ja) 2023-05-30
EA202092748A1 (ru) 2021-04-19
PH12020551995A1 (en) 2021-09-06
AU2019274461A1 (en) 2021-01-14
PE20211397A1 (es) 2021-07-27
US12168766B2 (en) 2024-12-17
IL278785B1 (en) 2024-11-01
CN112423792B (zh) 2024-04-23
KR20210011981A (ko) 2021-02-02
NI202000085A (es) 2021-03-11
EP3799570A1 (fr) 2021-04-07
KR20250153881A (ko) 2025-10-27

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